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Gene Therapy Cures Rare Disorder Affecting Dogs; Could Cure For Humans Be Next?

Update Date: Feb 16, 2017 08:06 PM EST

In the latest on Regenerative Medicine, researchers from University of Washington Medicine Institute for Stem Cell and Regenerative Medicine, have successfully cured a rare disorder affecting dogs through a gene therapy. The success of the gene therapy could very well translate into curing the same rare disorder affecting humans too.

According to the researchers, by being able to determine the correct dosage used in a gene therapy, the chances of survival of dogs affected with a rare disorder, previously known to be incurable, have helped restore the health of the affected dogs and prolonged their lives.

The rare disorder, an inherited neuromuscular disease, is due to the mutation of genes that usually synthesize a protein called myotubularin and is called myotubular myopathy or MTM.

MTM is a naturally occurring gene mutation that affects only the male species of dogs and humans. It causes the wasting of the muscles which usually leads to death. Patients, both canine and humans, affected with MTM are usually diagnosed during infancy and succumb to the rare disorder during their early lives due to difficulties in breathing brought upon by the disease.

For the past decades, various researchers have worked hard to find a suitable treatment for this kind of genetic muscle disease. Through the collaboration of different institutions, the researchers have finally found an effective treatment done by replacing the disease-causing MTM gene with a healthy gene.

The findings of the research, published in the journal Molecular Therapy, found that the safe gene therapy was able to cure the MTM-suffering dog. The dogs affected with myotubular myopathy, after receiving a single infusion of the corrective gene therapy, were unrecognizable from normal dogs a year after.

The success of the gene therapy in dogs could very well translate to the possible success of the gene therapy to humans afflicted with myotubular myopathy as well. Not only were the researchers able to find a cure but was also able to determine the appropriate dosage to be used in the gene therapy. The success of the gene therapy also demonstrates the potential of the treatment to possibly cure other diseases affecting both dogs and humans.

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